Catabasis Pharmaceuticals, Inc. reported new positive safety and efficacy results today, displaying the preservation of muscle function and sustained disease-modifying effects in patients with Duchenne muscular dystrophy (DMD) following 48 and 60 weeks of treatment with edasalonexent.
Consistent improvements in all assessments of muscle function were observed after more than a year of receiving 100 mg/kg/day of the potential oral foundational therapy in the Phase 2 MoveDMD trial and open-label extension, compared to the rates of change in the pre-specified control period for boys prior to receiving edasalonexent treatment. Catabasis has announced its intentions to initiate a single global Phase 3 trial with edasalonexent in patients with DMD, regardless of mutation type, in the first half of 2018. Top-line results are expected in 2020.
DMD is the result of a lack of functional dystrophin, the protein needed to keep muscle cells intact. Patients with the progressive degenerative muscle disorder – typically boys – experience symptoms in early childhood, and can lose the ability to walk by age 10.
As the disease continues to progress, these patients are prone to potentially fatal heart and lung complications.
“We are thrilled to see this preservation of muscle function and substantial slowing of disease progression in boys following more than a year of edasalonexent treatment. This effect has the potential to be extremely impactful for boys affected by Duchenne,” said Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis. “Building on the results previously reported for edasalonexent treatment in patients up to 36 weeks, these new data at 48 and 60 weeks show that edasalonexent continued to slow progression of the disease. We look forward to advancing edasalonexent in a single global Phase 3 trial later this year with the goal of improving the quality and length of life for those affected by Duchenne.”
Through 60 weeks of treatment in the Phase 2 and the open-label extension of the MoveDMD trial, the 100 mg/kg/day treatment group showed regular and clinically significant improvements in rates of decline compared to rates of change during the control period across all four assessments of muscle function. The assessments included three timed function tests (10-meter walk/run, 4-star climb, and time to stand), and the North Star Ambulatory Assessment (NSAA), which is a global assessment of muscle function.
In the same group, participants experienced positive effects of treatment via additional supportive measures of muscle health. Four muscle enzymes – creatine kinase, alanine aminotransferase, aspartate aminotransferase and lactate dehydrogenase – were significantly decreased compared to baseline following treatment at 12 and 60 weeks. This was consistent with previous data proving the drug’s ability to slow muscle degeneration and improve muscle integrity.
“As this study matures and we see the effect of edasalonexent treatment through 48 and 60 weeks, it is compelling that all of the assessments of muscle function are demonstrating stabilization at an age when boys with DMD have a predictable decline,” said Richard Finkel, M.D., Chief, Division of Neurology, Department of Pediatrics at Nemours Children’s Health System and a Principal Investigator for the study. “Clinically meaningful changes are observed here.”
To date, edasalonexent has been well-tolerated and no safety signals have been observed. Most adverse events (AEs) have been mild in nature, and the most common were primarily gastrointestinal.
For the upcoming Phase 3, Catabasis plans to enroll an estimated 125 patients who are between their 4th and 7th birthdays and have received steroids for at least 6 months.
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